{"id":"W4402969161","doi":"10.1016/j.omtn.2024.102349","title":"Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina","year":2024,"lang":"en","type":"article","venue":"Molecular Therapy — Nucleic Acids","topic":"CRISPR and Genetic Engineering","field":"Biochemistry, Genetics and Molecular Biology","cited_by":11,"is_retracted":false,"has_abstract":true,"ca_institutions":"","funders":"Domaine d’Intérêt Majeur Logiciels et Systèmes Complexes; Sorbonne Université; Fondation pour la Recherche Médicale; Institut National de la Santé et de la Recherche Médicale; AFM-Téléthon; Association Française contre les Myopathies; Agence Nationale de la Recherche; Fondation Voir et Entendre; Foundation Fighting Blindness","keywords":"Cas9; CRISPR; Genome editing; Computational biology; Guide RNA; Computer science; RNA; Base (topology); Retina; Genome; Biology; Genetics; Gene; Neuroscience","routes":{"ca_aff":false,"ca_fund":true,"ca_venue":false,"about_ca":false,"invisible_to_affiliation_only":true},"retraction":null,"screen":null,"direct_labels":[],"prediction":{"model_version":"metacan-v3-hybrid-931329e0061c","candidate_categories":[],"consensus_categories":[],"category_scores_codex":[0.0002449667,0.0003367722,0.0003348942,0.0001553459,0.0002402398,0.0005685125,0.0002736108,0.0006617498,0.0013193],"category_scores_gemma":[0.0003020105,0.000171064,0.0002199165,0.00008897821,0.0002916647,0.0002729758,0.000345846,0.0005749221,0.0006235981],"about_ca_system_candidate":false,"about_ca_system_consensus":false,"about_ca_system_score_codex":0.0003543338,"about_ca_system_score_gemma":0.0003318081,"about_ca_topic_candidate":false,"about_ca_topic_consensus":false,"about_ca_topic_score_codex":0.0008467109,"about_ca_topic_score_gemma":0.001452906,"domain_scores_codex":[0.9996703,0.00004045686,0.00002658803,0.0001086093,0.0001251497,0.00002892074],"domain_scores_gemma":[0.9998477,0.00004713739,0.00004543242,0.00002152867,0.00001634816,0.00002189338],"domain_codex":null,"domain_gemma":null,"domain_candidate":null,"domain_consensus":null,"study_design_codex":"bench_or_experimental","study_design_gemma":"bench_or_experimental","study_design_scores_codex":[0.00001840174,0.000009579217,0.0001039523,0.00004066893,0.000005717808,0.00004819961,0.00001024575,0.00006839187,0.9970359,0.0002736688,0.00004174158,0.002343627],"study_design_scores_gemma":[0.000003982809,0.0001045622,0.0004515219,0.000004586359,0.000009893392,0.0002159503,0.000009600478,0.0007142337,0.9948597,0.00005383088,0.003567977,0.000004163012],"study_design_candidate":"bench_or_experimental","study_design_consensus":"bench_or_experimental","genre_codex":"empirical","genre_gemma":"empirical","genre_scores_codex":[0.8954546,0.00702262,0.08397414,0.000444978,0.0001809884,0.0001908456,0.0003407784,0.0007383115,0.01165281],"genre_scores_gemma":[0.952483,0.00227665,0.0348174,0.0001131083,0.00002709088,0.00004990898,0.0002863249,0.0000946669,0.009851816],"genre_candidate":"empirical","genre_consensus":"empirical","teacher_disagreement_score":0.0013193,"threshold_uncertainty_score":0.004413486,"prediction_status":"machine_predicted_unvalidated"},"machine_scores":{"provisional":true,"baseline":true,"maturity_gate_passed":false,"score_opus":0.01028850280644513,"score_gpt":0.2708784636405886,"score_spread":0.2605899608341435,"validation_status":"score_only:v0-immature-baseline","note":"Baseline scores from an immature model (maturity gate not passed). Scores rank; they never assert a category."}}